Trial · NCT02603562
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
NCT02603562, Phase 1, Phase 2, Completed, aTyr Pharma, Inc.; results first posted 2023-10-19
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy. Trials, record NCT02603562. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02603562
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
- NCT number
- NCT02603562
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 8 participants, actual
- Lead sponsor
- aTyr Pharma, Inc.
- Conditions
- Facioscapulohumeral Muscular Dystrophy (FSHD)
- Interventions
- ATYR1940
- First posted
- 2015-11-13
- Last updated
- 2023-10-19
- Other registries' IDs
- eudract:2014-003346-27
- Results first posted
- 2023-10-19
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to End of Study (up to Week 25) | TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section. |
| Primary | Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | Up to End of Study (Up to Week 25) | Laboratory parameters included hematology (hematocrit, hemoglobin, red blood cell count, white blood cell count with differential \[neutrophils, lymphocytes, monocytes, eosinophils, basophils\], and platelet count); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase, alkaline phosphatase, total protein, sodium, potassium, bicarbonate, calcium, chloride, magnesium, inorganic phosphate, creatine kinase, lactate dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, and cholesterol \[nonfasting\]); and urinalysis (color, pH, specific gravity, protein, glucose, ketones, and blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With an Ocular Abnormality Leading to a TEAE | Up to End of Study (Up to Week 25) | Ocular parameters included vitreous, retina, macula, choroid, optic nerve, and optic nerve pallor. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Primary | Number of Participants With an Impact on Hearing From ATYR1940 Treatment | Up to End of Study (Up to Week 25) | |
| Primary | Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | Up to End of Study (up to Week 25) | Pulmonary evaluations included pulmonary function tests and pulse oximetry. Clinically significant changes were to be reported as adverse events. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Baseline up to Week 12 | Titers through Week 12 are summarized. |
| Secondary | Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | Baseline up to Week 12 | Participants with Jo-1 Ab levels ≥1.5 units/milliliter (U/mL) were to be discontinued from dosing of the study drug. |
| Secondary | Number of Participants With Infusion-Related Reactions | Baseline up to Week 12 | Infusion-related reactions included fever, chills, rigors, myalgia, facial erythema, systemic erythema, pallor, facial swelling, chest tightness, difficulty breathing, wheezing, stridor, tachypnea, bronchospasm, cough, tachycardia, significant pulse rate increase from baseline without obvious cause, bradycardia, significant pulse rate decrease from baseline, pre-syncope or syncope, hypotension, orthostatic hypotension, blood pressure swings (including hypertension), skin rash, urticaria, angioedema, pruritus, difficulty speaking, hoarse voice, raspy voice, excessive salivation, difficulty swallowing, nausea, vomiting, cramps, diarrhea; swelling of the throat, tongue, mouth, or lip, and development of a headache especially moderate or greater after start of infusion. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Secondary | Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 14 | Baseline, Week 14 | MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted-MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Up to End of Study (up to Week 25) | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | Up to End of Study (Up to Week 25) | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With an Ocular Abnormality Leading to a TEAE | Up to End of Study (Up to Week 25) | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With an Impact on Hearing From ATYR1940 Treatment | Up to End of Study (Up to Week 25) | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | Up to End of Study (up to Week 25) | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Positive Anti-Drug Antibodies (ADA) | Baseline up to Week 12 | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | Baseline up to Week 12 | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Infusion-Related Reactions | Baseline up to Week 12 | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 14 | Baseline, Week 14 | Safety analysis set included all participants who received at least 1 full or partial dose of ATYR1940 and had a post-infusion safety observation. | percent change | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | ATYR1940 | TEAEs | 8 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | ATYR1940 | SAEs | 0 | Participants | |
| Number of Participants With a Clinical Laboratory Abnormality Leading to an AE | ATYR1940 | 1 | Participants | ||
| Number of Participants With an Ocular Abnormality Leading to a TEAE | ATYR1940 | 0 | Participants | ||
| Number of Participants With an Impact on Hearing From ATYR1940 Treatment | ATYR1940 | 0 | Participants | ||
| Number of Participants With a Clinically Significant Pulmonary Function Event Resulting in a TEAE | ATYR1940 | 0 | Participants | ||
| Number of Participants With Positive Anti-Drug Antibodies (ADA) | ATYR1940 | 4 | Participants | ||
| Number of Participants With a Jo-1 Antibody (Ab) Test Result ≥1.5 Units/Milliliter (U/mL) | ATYR1940 | 0 | Participants | ||
| Number of Participants With Infusion-Related Reactions | ATYR1940 | 1 | Participants | ||
| Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 14 | ATYR1940 | 3.8 | Full Range -6 to 19 | percent change |
Source ClinicalTrials.gov
Participant flow4
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | ATYR1940 | 8 | |
| Overall Study | Received at Least 1 Dose of Study Drug | ATYR1940 | 8 | |
| Overall Study | COMPLETED | ATYR1940 | 7 | |
| Overall Study | NOT COMPLETED | ATYR1940 | 1 |
Source ClinicalTrials.gov
Adverse events29
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Paraesthesia | Nervous system disorders | ATYR1940 | 3 | 8 | |
| other | Headache | Nervous system disorders | ATYR1940 | 2 | 8 | |
| other | Nausea | Gastrointestinal disorders | ATYR1940 | 2 | 8 | |
| other | Myalgia | Musculoskeletal and connective tissue disorders | ATYR1940 | 3 | 8 | |
| other | Nasopharyngitis | Infections and infestations | ATYR1940 | 2 | 8 | |
| other | Muscular weakness | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Musculoskeletal pain | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Pain in extremity | Musculoskeletal and connective tissue disorders | ATYR1940 | 1 | 8 | |
| other | Ageusia | Nervous system disorders | ATYR1940 | 1 | 8 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | ATYR1940 | ||
| Total, other adverse events | ATYR1940 | 8 | 8 |
| Total, serious adverse events | ATYR1940 | 0 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02603562
GET https://conductscience.org/api/trials/v1/NCT02603562/events?page=2